Gene Therapy For Neuromuscular Disorders

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1. Gene therapy 1.1.1. Goal of gene replacement therapy for neuromuscular disorders. • What is gene therapy Definition: Gene therapy is the introduction of nucleic acid, including DNA, RNA and their analogue into cells of living organism to treat diseases. This is occurred through modifying expression of gene of interests, triggering alteration of certain biological functions. The target of gene therapy is confined to living cells, mostly because that the native DNA and RNA processing machinery is needed to amplify the introduced genetic information and generate functional products, such as protein, shRNA or microRNA. To a lesser extent, gene therapy aims at modulating the function of recipient cells. The exception here is the secreted molecules, such as clotting factors. As a result, the abundance of target cells, to certain extent, determines the effect of gene therapy. This is especially true for myopathy. In the advanced stage of diseases, such as DMD, surviving myofibers are so limited that even the function of individual myofiber is fully restored, there would be no appreciable functional improvement on tissue level. The advent of stem cell technology, including human embryonic stem cells and induced pluripotent stem cells (iPSCs) has the potential to overcome this bottleneck of gene therapy. Because of its indefinite self-replicating potential and the capacity to be converted into nearly all cell types within the body, pluripotent stem cells can theoretically
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